Sentences with phrase «haemophilia patients»
The results, published in the New England Journal of Medicine, demonstrate the therapy being used to treat 10 male haemophilia patients without significant side effects.
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Ghosh's research leads to the conclusion that a patient with haemophilia who co-inherits a thrombophilic gene bleeds less than one without that mutation.
But many of the physicians who treat patients with haemophilia are not convinced.
«These are remarkable data,» says Amit Nathwani at University College London, who is using AAVs in potential treatments for a blood disorder called haemophilia B. «Liver fibrosis is a major clinical problem and if these data can be reproduced clinically, the National Health Service would save billions and patients would be given a new lease of life.»
Indeed, exposure of the protein produced by the nanoparticle - based gene therapy to the gut mucosa prevents inhibitor development and restores clotting - factor activity in mouse models of both haemophilia A and B. «This approach really could hold big benefit for patients,» says Jörg Schüttrumpf, a transfusion - medicine specialist who led one of the studies performed at the German Red Cross Blood Donor Service in Frankfurt.
Some concerns had been raised that by infusing patients with such a high activity gene, the treatment would overcorrect for haemophilia and give rise to thrombosis.
Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, after being tested in 10 male patients
Spark Therapeutics» gene therapy for patients with haemophilia has seen some early success in clinical trials, according to a report published yesterday.